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Avidity Biosciences, Inc.`s mission is to profoundly improve people`s lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity`s proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity`s advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 (DM1) and is currently in Phase 1/2 development with the ongoing MARINA™ and MARINA-OLE™ trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy (FSHD) and is currently in Phase 1/2 development with the FORTITUDE™ trial. AOC 1044 is designed for people with Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44™ trial. AOC 1044 is the first of multiple AOCs the company is developing for DMD. Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in San Diego, CA.
Geltor is the bio-design company that makes the world`s most advanced proteins for consumers, with a focus on unique functionality and sustainability.
Diasome Pharmaceuticals, Inc. is focused on the clinical and commercial development of breakthrough therapies for diabetes and obesity.
Sera Prognostics is a leading proteomic and bioinformatics company dedicated to improving the lives of women and babies through precision biomarker-based tests designed to enhance pregnancy care. Sera’s vision is to deliver pivotal information in early pregnancy to physicians, to help them to improve the health of their patients and reduce costs of healthcare delivery. Sera’s PreTRM® Test reports to a physician the individualized risk of a pregnant woman to deliver prematurely, enabling earlier proactive interventions in patients with higher risk. Rigorous clinical validation of PreTRM® Test performance (accuracy of predicting premature delivery) was reported in the American Journal of Obstetrics & Gynecology in 2016 in a U.S. cohort of 5,501 patients across 11 centers. Preterm birth is defined as any birth before 37 weeks gestation and is a leading cause of illness and death in newborns. The 2020 March of Dimes Report Card shows that of nearly 4 million babies born annually in the U.S., more than one in ten is born prematurely. Prematurity is associated with a significantly increased risk of major long-term medical complications, including learning disabilities, cerebral palsy, chronic respiratory illness, intellectual disability, seizures, vision and hearing loss, and can generate significant cost throughout the lives of affected children. The annual U.S. health care costs to manage complications of prematurity were estimated at $31.5B for 2015. Sera is also developing a robust pipeline of innovative tests focused on other complications of pregnancy. Using its advanced proprietary mass spectrometry and bioinformatics platform technologies, Sera detects biologically important protein expression differences to build high performing predictions of risk for adverse pregnancy outcomes (including preterm birth, preeclampsia, gestational diabetes, growth restriction, and others).
Vital Therapies, Inc. is a biotherapeutic company focused on developing a cell-based system for the treatment of acute liver failure. Our product candidate, the ELAD® System, is a human cell-based, bio-artificial liver support system that operates outside the body, or extracorporeally, and is designed with the proposed intent to allow the patient’s own liver to regenerate to a healthy state, or to stabilize the patient until liver transplant. The ELAD System incorporates our human liver-derived cells, or VTL C3A cells, contained in four hollow fiber cartridges, that are combined with single use customized disposable sets and an ancillary delivery system. Data from ELAD clinical studies has shown trends that may indicate a potential to increase survival rates in patients with acute liver failure. ELAD has received orphan designation in the United States and Europe for the treatment of acute liver failure. Prior to the initiation of our ongoing Phase III clinical trial program, over 145 subjects have received therapy with the ELAD System in seven clinical trials and through a compassionate use program, which we believe collectively suggests a promising therapeutic profile. In March 2013, we initiated VTI-208, a Phase III randomized, controlled clinical trial in 200 subjects with alcohol-induced liver decompensation. We reached the midpoint in enrollment of this trial in April 2014, and anticipate the release of preliminary data in the first half of 2015. In addition, we are conducting a second Phase III randomized, controlled clinical trial, VTI-210, in 150 subjects with severe acute alcoholic hepatitis, or AAH, which is a subset of AILD, and expect to initiate enrollment of subjects later in 2014. In the second quarter of 2014, we began enrollment of a Phase II clinical trial of the ELAD System in subjects with either fulminant hepatic failure, or FHF, or surgery-induced acute liver failure, or SILF. We anticipate the release of data from VTI-210 in 2016 and the Phase II component of VTI-212 in 2015 or 2016.